The European Commission has granted marketing authorization to Shire plc (Cambridge, MA) for VPRIV (velaglucerase alfa), a human cell line–derived enzyme replacement therapy for the long-term treatment of type 1 Gaucher disease. VPRIV has been authorized as an orphan drug, making it available in 30 countries across Europe.
This approval was based on data from Shire's velaglucerase alfa clinical development program for type 1 Gaucher disease. Over 100 Gaucher patients at 24 sites in 10 countries around the world participated in the clinical studies, all of which met their primary endpoints.
Earlier this year, the US Food and Drug Administration approved VPRIV following priority review to treat children and adults with type 1 Gaucher disease in the United States. VPRIV is an alternative to Cerezyme (imiglucerase), a drug manufactured by Genzyme (Cambridge, MA). Cerezyme currently is in short supply because of manufacturing problems at the Genzyme plants that produce Cerezyme. Because of the Cerezyme shortage, Shire also had received an FDA-approved treatment protocol to allow early patient access to velaglucerase alfa before it was approved.
FDA Approves Shire's New Gaucher Drug
Genzyme Expects $175 Million Fine